From Satellites To Pharmaceuticals

In 1990, 6-year-old Jenesis Rothblatt's lips turned blue. The little girl gasped for breath, could not climb stairs and was soon in the intensive care unit of Children's National Hospital in Washington, D.C.

The disaster began a transformation for Jeni's father--and a change in the way drugs for rare diseases are developed. Martin Rothblatt, a lawyer and businessman, turned his childhood passion for outer space into founding a series of companies that made him millions of dollars. First came GeoStar, a GPS-based navigation system, in 1986, then an early satellite radio company called Worldspace and finally
Sirius Satellite Radio
in 1990. In 1994 he underwent gender reassignment surgery and changed his name to Martine. Now a she, 55, Martine remains married to Jeni's mom.

When Jeni became sick, Martine felt useless. "I was an expert in satellites, and I didn't know anything about medicine," she says. Jeni had pulmonary arterial hypertension (PAH), a rare and fatal disease that is caused when the artery between the heart and lungs is damaged. With 200,000 cases worldwide, PAH was too uncommon to win drug-development money. Martine Rothblatt sold her telecom stock and started a $3 million foundation to fund research, but three years went by without any progress.

One of her daughter's doctors told her about a potential treatment that was languishing at Glaxo Wellcome. A banker friend started feeding her analyst reports on biotechnology companies. What she had done with satellites, she reasoned, she could also do with drugs. She just needed to persuade some investors to pony up.
United Therapeutics
was founded in Silver Spring, Md. in 1996.

Initially investors were skeptical. How would a tiny population of patients support a drug company? But by the mid-1990s the first examples of orphan drugs had hit the market.
Genzyme
, of Cambridge, Mass., had been charging $150,000 a year for a treatment for a far rarer disease than PAH (Gaucher's disease), but large drug companies still saw rare diseases as small opportunities. Viagra showed early efficacy in PAH, but it was its use in erectile dysfunction that got
Pfizer
excited. Martine used her M.B.A. and calculated that a drug for PAH would have to cost $100,000 a year to support its development. Then, ever the lawyer, she looked at her own health plan. It limited the total amount it would pay for medical treatments over a lifetime, but there was no limit on how much a drug could cost per dose. With help from a Glaxo executive whose sister had PAH, Rothblatt was able to license Glaxo's drug.

Everybody else in biotech had a Ph.D. or an M.D., so Martine decided to get one, too. The late John Vane, a 1982 Nobel laureate in medicine who was advising United Therapeutics, hooked Rothblatt up with bioethicists at the University of London. At the time there was a raging controversy over efforts by
Novartis
to create genetically modified pigs whose lungs and other organs could be transplanted into people. Some virologists worried that retroviruses in the pigs could be transmitted to people via the transplants. This infuriated Rothblatt, because those lungs could save the lives of PAH patients who had run out of options. She wrote her thesis on how the research could be ethically conducted and earned her Ph.D. in bioethics in 2001. Pig-to-human organ transplant research remains banned.

The Food & Drug Administration, however, was not all that enthused about United Therapeutics' drug, by then dubbed Remodulin. The drug is a synthetic version of natural chemicals called prostacyclins that cause blood vessels to open. Results in clinical trials were not all that much better than for Flolan, a competing drug, FDA reviewers argued. Remodulin was delayed for two years, but the drug was finally approved in 2002 after a second study and priced at $90,000 per year. Within a year annual sales were $50 million, rising to $300 million last year. United Therapeutics shares are up 800% since the company went public in 1999 and have doubled in the past year because of hopes for a new inhaled version of Remodulin and a deal with
Eli Lilly
to sell the erectile-dysfunction drug Cialis as an alternative treatment for PAH. Some analysts worry that the inhaled drug, Tyvaso, will cannibalize Remodulin sales, but Rothblatt says there is a market, since Remodulin currently must be injected.

At first telling investors that she was going to charge a high price for a treatment for people like her daughter gave Rothblatt "a sour taste" in her mouth. But the availability of treatments changed the nature of PAH. There were 75 specialists in PAH in the U.S. when Jeni got sick. Now 10,000 doctors treat it. Pfizer, Actelion and
Gilead Sciences
all market pills for PAH. The disease is still invariably fatal, but patients are living much longer, in some cases decades after diagnosis.

In 2004 Rothblatt and her wife, Bina, cofounded a nonprofit, the Terasem Movement, dedicated to promoting the idea that science can radically extend the human life span. She says that medicine will allow human beings to "transcend commonly accepted limits" of human life and calls futurist Raymond Kurzweil, who promotes similar ideas, "the smartest guy on the planet." Kurzweil also serves on United Therapeutics' board of directors.

Jeni Rothblatt, now 26, doesn't disclose what medicines she takes, but she is doing fine. She works at United Therapeutics, setting up town halls in the online virtual world of Second Life, where Martine can interact with all 410 of her employees, whom she calls Unitherians.

Rothblatt has made some investors uneasy by selling stock; she says much of her net worth is still tied up in United. Jonathan Aschoff, the biotech analyst at investment bank Brean Murray Carret, expects strong sales and late-stage results of an oral Remodulin formulation to goose the stock. Things get tougher when the Remodulin patent expires in 2014. That leaves plenty of time for Rothblatt to find the next big PAH drug.